Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.

 

Posted on: 15 August 2022, source: genengnews.com
Novartis has acknowledged that two patients have died of acute liver failure following treatment with its Zolgensma® (onasemnogene abeparvovec-xioi), a one-time gene therapy indicated for some forms of spinal muscular atrophy (SMA). As a result, the company said, it will revise Zolgensma’s label to specify that fatal acute liver failure has been reported.

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Posted on: 21 July 2022, source: LUMC
Researchers from the Leiden University Medical Center (LUMC) have successfully used stem cell gene therapy to treat a baby with the severe congenital immune disorder SCID. An important milestone: it is the first time stem cell gene therapy of Dutch origin has been administered to a patient, and also the first time it has been used to treat this particular form of SCID worldwide. The treatment was effective and the patient is doing well.

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Posted on: 12 July 2022, source: ohsu.edu
A new pre-clinical study in nonhuman primates will evaluate an experimental drug’s potential use as a gene therapy that could prevent people who have HIV from having to take daily antiviral drugs for the rest of their lives. The research will be led by Oregon Health & Science University researcher Jonah Sacha, Ph.D., who also serves as a scientific adviser to CytoDyn, the biotechnology company developing the drug, called leronlimab. The study is funded by a five-year grant of up to $5 million that was recently awarded to OHSU by the National Institute of Allergy and Infectious Disease, which is part of the National Institutes of Health.

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Posted on: 13 June 2022, source: Business Standard
ImmunoACT, an IIT-Bombay spin-off backed by Hyderabad-based Laurus Labs, is testing a new gene-therapy treatment for cancer, which they claim would not only cure the disease, but also costs one-tenth of the global price. “We are working with CD19 CAR T Cell for leukemia and lymphoma. What we do is we take out some blood and plasma from the cancer patient, and then isolate the T-cells (which are immune cells) from this sample. These cells are then genetically modified so that they eliminate cancer cells. Then this is transfused back to the patient,” explains Rahul Purwar, founder and chairman of ImmunoACT. The product is named H-CAR T-19.

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Posted on: 31 May 2022, source: SciTechDaily
In mouse studies, pain-blocking neurotransmitters produced long-lasting benefits without detectable side effects. An international team of researchers led by scientists at the University of California San Diego School of Medicine reported that a gene therapy that inhibits targeted nerve cell signaling effectively reduced neuropathic pain in mice with spinal cord or peripheral nerve injuries with no detectable side effects.

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Posted on: 15 May 2022, source: ASGCT
St. Jude Children’s Research Hospital scientists will discuss research on cellular therapies for pediatric cancer and more at the annual gathering of the professional society for gene and cellular therapy researchers.

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Posted on: 15 April 2022, source: Hansonwade
Inconsistent long-term clinical data has highlighted the need to better understand the durability of AAV gene therapy. At Hanson Wade Intelligence, our Market Research division, we have classified 121 AAV gene therapy trials into 4 distinct groups based on the target tissue type – liver, muscle, CNS and ocular tissues. This allowed us to summarise available data on clinical durability as well as the key findings we observed across tissues, trials, doses and serotypes. We hope that this report will provide gene therapy developers with valuable insights on the durability of the current generation of AAV gene therapies.

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